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Treatment of pediatric FH is achieved through lifestyle modifications, lipid\u2010modifying pharmacotherapy, and\/or apheresis. The primary objective of this review is to describe the characteristics of clinical trials conducted in pediatric patients with FH with data submitted to the US Food and Drug Administration from 2007 to 2020. Of 10 trials with 8 products in pediatric FH submitted to the Food and Drug Administration, 1 product was studied in both the heterozygous and the homozygous phenotypes, 5 were studied for heterozygous hypercholesterolemia only, and 2 were studied for homozygous familial hypercholesterolemia only. Most of the trials included pediatric patients \u226510 years of age and older. Clinical trial characteristics including the primary efficacy end points between pediatric and adult trials were mostly identical. Many lipid\u2010lowering drugs with novel mechanisms of action have been recently approved or are currently being studied. In summary, the drug treatment of hypercholesterolemia in pediatric patients is expanding beyond the use of statins, and now involves multiple mechanisms of action involving cholesterol metabolism. As younger pediatric patients are diagnosed and treated for heterozygous familial hypercholesterolemia and homozygous familial hypercholesterolemia, optimizing the doses of these agents and safety studies specific to younger pediatric patients will be necessary.<\/jats:p>","DOI":"10.1002\/jcph.1973","type":"journal-article","created":{"date-parts":[[2021,9,25]],"date-time":"2021-09-25T14:20:17Z","timestamp":1632579617000},"page":"397-408","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":5,"title":["Pediatric Drug Development Studies for Familial Hypercholesterolemia Submitted to the US Food and Drug Administration Between 2007 and 2020"],"prefix":"10.1002","volume":"62","author":[{"given":"Kyunghun","family":"Park","sequence":"first","affiliation":[{"name":"Office of Clinical Pharmacology Center for Drug Evaluation and Research US Food and Drug Administration  Silver Spring Maryland USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kristina","family":"Vishnevetskaya","sequence":"additional","affiliation":[{"name":"University of North Carolina Eshelman School of Pharmacy  Chapel Hill North Carolina USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jayabharathi","family":"Vaidyanathan","sequence":"additional","affiliation":[{"name":"Office of Clinical Pharmacology Center for Drug Evaluation and Research US Food and Drug Administration  Silver Spring Maryland USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gilbert J.","family":"Burckart","sequence":"additional","affiliation":[{"name":"Office of Clinical Pharmacology Center for Drug Evaluation and Research US Food and Drug Administration  Silver Spring Maryland USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dionna J.","family":"Green","sequence":"additional","affiliation":[{"name":"Office of Pediatric Therapeutics Commissioner's Office US Food and Drug Administration  Silver Spring Maryland USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2021,10,19]]},"reference":[{"key":"e_1_2_9_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jacl.2015.04.009"},{"key":"e_1_2_9_3_1","first-page":"49","article-title":"Familial hypercholesterolaemia","volume":"25","author":"Marais AD","year":"2004","journal-title":"Clin Biochem Rev"},{"key":"e_1_2_9_4_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jacc.2020.03.057"},{"key":"e_1_2_9_5_1","doi-asserted-by":"publisher","DOI":"10.1136\/bmjopen-2017-016461"},{"key":"e_1_2_9_6_1","doi-asserted-by":"publisher","DOI":"10.1093\/eurheartj\/ehv157"},{"key":"e_1_2_9_7_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jacl.2011.04.003"},{"key":"e_1_2_9_8_1","doi-asserted-by":"publisher","DOI":"10.4158\/EP171764.GL"},{"key":"e_1_2_9_9_1","doi-asserted-by":"publisher","DOI":"10.1542\/peds.2008-1349"},{"key":"e_1_2_9_10_1","doi-asserted-by":"publisher","DOI":"10.1161\/CIR.0000000000000618"},{"key":"e_1_2_9_11_1","first-page":"e1082","article-title":"2018 AHA\/ACC\/AACVPR\/AAPA\/ABC\/ACPM\/ADA\/AGS\/APhA\/ASPC\/NLA\/PCNA guideline on the management of blood cholesterol: a report of the American College of Cardiology\/American Heart Association Task Force on Clinical Practice Guidelines","volume":"139","author":"Grundy SM","year":"2019","journal-title":"Circulation"},{"key":"e_1_2_9_12_1","unstructured":"US Food and Drug Administration. 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